EU/3/20/2257 - orphan designation for treatment of peripheral T-cell lymphoma

Sintilimab

Overview

On 28 February 2020, orphan designation EU/3/20/2257 was granted by the European Commission to Parexel International GmbH, Germany, for sintilimab for the treatment of peripheral T-cell lymphoma.

What is peripheral T-cell lymphoma?

Peripheral T-cell lymphoma is a cancer of the lymphatic system, a network of vessels that transport fluid from tissues through the lymph nodes and into the bloodstream. In peripheral T-cell lymphoma there is uncontrolled growth of T lymphocytes (T cells), a type of white blood cell found in the lymphatic system. Peripheral T-cell lymphomas include types that mainly occur in the lymph nodes (primary nodal) and types that occur mainly outside the lymph nodes (primary extranodal).

The symptoms of the disease vary according to the type of lymphoma, but the first sign may be a lump in the neck, under the arm or in the groin area, which is caused by an enlarged lymph node. The lymphoma may also affect other organs in the body such as the bone marrow, liver and the skin.

Peripheral T-cell lymphoma is a long-term debilitating and life-threatening condition because in most cases the disease does not respond well to therapy, usually comes back within one year and is associated with early death.

What is the estimated number of patients affected by the condition?

At the time of designation, peripheral T-cell lymphoma affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 52,000 people^*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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^*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 519,200,000 (Eurostat 2020).

 

What treatments are available?

At the time of designation, the main treatment for peripheral T-cell lymphoma was chemotherapy (medicines to treat cancer), sometimes in combination with radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with peripheral T-cell lymphoma because early studies showed that the medicine can be of benefit in patients whose disease had not improved with previous treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Peripheral T-cell lymphoma cells produce proteins (PD-L1) that switch off cancer-killing cells of the immune system called T cells by attaching to a target on the T-cells called PD-1. This prevents the T cells from attacking the cancer. Sintilimab is a monoclonal antibody, a type of protein, designed to attach to PD-1 without switching the T cells off itself. This blocks the cancer cell proteins from attaching and switching the cells off, thereby increasing the ability of the immune system to kill cancer cells.

What is the stage of development of this medicine?

The effects of sintilimab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with sintilimab in patients with peripheral T-cell lymphoma were ongoing.

At the time of submission, sintilimab was not authorised anywhere in the EU for the treatment of peripheral T-cell lymphoma or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 22 January 2020, recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/20/2257: Public summary of positive opinion for orphan designation of sintilimab for the treatment of peripheral T-cell lymphoma

Reference Number: EMADOC-628903358-1938

Key facts

• Active substance:
  Sintilimab
• Intended use:
  Treatment of peripheral T-cell lymphoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/20/2257
• Date of designation:
  28/02/2020
• Sponsor:

  Parexel International GmbH;